BOSTON—
Working toward broadening its pipeline and acquiring
future innovative therapies, global specialty biopharmaceutical Shire PLC
recently made two significant investments in potentially innovative treatment
protocols. In early March, the company agreed to buy privately held FerroKin
BioScience for an upfront payment of $100 million, plus potential milestone
payments of up to $225 million, in an effort to boost its
hematology business.
In addition, Shire also purchased the worldwide exclusive license for the
development and commercialization of an adenosine A2A
antagonist from United
Kingdom-based Heptares to treat central
nervous system (CNS) disorders.

Shire was strongly attracted to Ferrokin's lead product, a
potential treatment for iron overload that is being studied in Phase II
clinical studies in patients who have received multiple blood transfusions.

In the agreement with Heptares, Shire has agreed to pay the
company an upfront option grant, plus future development and
commercial
milestone payments that could total $190 million, as well as royalties on
product sales. Further terms of the agreement were not disclosed.

Jeff Jonas, senior vice president of research and
development for Shire's Specialty
Pharmaceuticals and Regenerative Medicine
unit, says Shire is continuously searching for innovations that have the
potential to help the company
develop differentiated medicines.

"This agreement with Heptares is a reflection of our growth
strategy of investing and focusing on highly targeted drug discovery
platforms," Jonas says. "We are impressed with the novelty and quality of the
A2A antagonist leads generated by Heptares, resulting from what we believe to
be the first time a structure-based drug discovery approach has been
applied
from the beginning to a GPCR drug target."

Currently in preclinical development,
adenosine A2A is a
G-protein coupled receptor (GPCR) involved in the regulation of dopaminergic
pathways in the brain. Inhibition of the A2A receptor
is a validated mechanism
in the treatment of CNS disorders, but Heptares has taken it one giant step
farther by actually discovering and developing new
medicines targeting GPCRs,
which are linked to a wide range of human diseases.

Adenosine A2A, in
fact, could eventually prove to be a new
treatment for a range of CNS disorders. While the specific CNS disease targets
have not been disclosed,
similar rival drugs are already in development to
treat Parkinson's disease.

Malcolm Weir, CEO of Heptares, tells ddn, "From Heptares' perspective, Shire is one of the world's
leading
CNS specialty pharmaceutical companies—and the fit of product
opportunity and company cultures was particularly good. Heptares aims to be the
leading
company that discovers and develops new medicines targeting GPCRs, and
it believes its unique approach and capabilities will enable this through the
development of a broad pipeline of drug candidates for serious CNS and
metabolic disorders, internally balanced with strategic alliances with pharma
partners. The upfront fees and potential profits will be used to execute the
company's strategy and grow Heptares through its structure-based drug
discovery
capabilities and advancement of its internal pipeline."

The
major problems with CNS therapies are lack of efficacy
and side effects that result from non-specific binding of sub-optimal small
molecule drugs, he
notes. Many CNS disorders can be traced back to GPCR
activity. GPCRs are key in signal transduction pathways.

"Heptares has overcome this major obstacle," Weir said. "Its technology
can stabilize GPCRs in pharmacologically relevant conformations and generate
detailed structural information that can enable drugs to be designed
atom-by-atom, thus offering high potency and specificity, fewer side effects
and
potential first-in-class or best-in-class drug opportunities."

DUBLIN, Ireland—Shire PLC announced April 12 that it has
signed an agreement to acquire substantially all the
assets of Pervasis
Therapeutics, for an undisclosed sum.

Per the company's announcement, Shire will provide Pervasis
with an upfront payment in addition
to potential post-closing milestone
payments that are dependent on Shire's achievement of certain clinical
development, regulatory and sales targets.

According to Shire, the acquisition complements its 2011
purchase of Advanced BioHealing and
further demonstrates Shire's commitment to
investing in and building out its portfolio of regenerative medicine therapies.

Pervasis' clinical development assets bring to Shire a new
cell-based technology platform—endothelial cell technology—with
potential
global opportunity. The company's lead program, Vascugel, addresses a
significant unmet medical need for acute vascular repair technologies
focused
on improving hemodialysis access for patients with end-stage renal disease
(ESRD). With diabetes being the global leading cause of ESRD, and
accounting
for approximately 40 percent of ESRD patients in the United States, Pervasis'
Vascugel therapy complements ABH's Dermagraft, which is
indicated for diabetic
foot ulcers, a condition highly prevalent among diabetic dialysis patients.

Shire will initially focus on completing a Phase II program
to address maturation and maintenance of arteriovenous (AV) access for
hemodialysis
patients, which will instruct a future development pathway.

"There are currently no approved
therapies that directly target
the underlying physiological processes associated with the creation of AV
access sites in hemodialysis patients,
including inflammation, thrombosis, and
restenosis," said Shire Regenerative Medicine President Kevin Rakin in a
statement. "As a result, there
remains a significant unmet need for
technologies that improve hemodialysis access for patients with ESRD. We
believe Vascugel has the potential to
enhance the rate of blood vessel repair
while also providing for increased maintenance of the access site for a
prolonged period of time as compared to
current treatments. This acquisition
marks a very important step for Shire in building a Regenerative Medicine
business focused on tissue repair and
regeneration."